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Page 1
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M. Liu G, et al. Among authors: weetall m. Hum Mol Genet. 2024 Jan 20;33(3):211-223. doi: 10.1093/hmg/ddad173. Hum Mol Genet. 2024. PMID: 37819629 Free PMC article.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Among authors: weetall m. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Structure-activity relationship (SAR) optimization of 6-(indol-2-yl)pyridine-3-sulfonamides: identification of potent, selective, and orally bioavailable small molecules targeting hepatitis C (HCV) NS4B.
Zhang N, Zhang X, Zhu J, Turpoff A, Chen G, Morrill C, Huang S, Lennox W, Kakarla R, Liu R, Li C, Ren H, Almstead N, Venkatraman S, Njoroge FG, Gu Z, Clausen V, Graci J, Jung SP, Zheng Y, Colacino JM, Lahser F, Sheedy J, Mollin A, Weetall M, Nomeir A, Karp GM. Zhang N, et al. Among authors: weetall m. J Med Chem. 2014 Mar 13;57(5):2121-35. doi: 10.1021/jm401621g. Epub 2013 Dec 4. J Med Chem. 2014. PMID: 24266880
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Among authors: weetall m. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
6-(Azaindol-2-yl)pyridine-3-sulfonamides as potent and selective inhibitors targeting hepatitis C virus NS4B.
Chen G, Ren H, Zhang N, Lennox W, Turpoff A, Paget S, Li C, Almstead N, Njoroge FG, Gu Z, Graci J, Jung SP, Colacino J, Lahser F, Zhao X, Weetall M, Nomeir A, Karp GM. Chen G, et al. Among authors: weetall m. Bioorg Med Chem Lett. 2015 Feb 15;25(4):781-6. doi: 10.1016/j.bmcl.2014.12.093. Epub 2015 Jan 7. Bioorg Med Chem Lett. 2015. PMID: 25613678
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M. Zhao X, et al. Among authors: weetall m. Hum Mol Genet. 2016 May 15;25(10):1885-1899. doi: 10.1093/hmg/ddw062. Epub 2016 Feb 29. Hum Mol Genet. 2016. PMID: 26931466 Free PMC article.
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. Woll MG, et al. Among authors: weetall m. J Med Chem. 2016 Jul 14;59(13):6070-85. doi: 10.1021/acs.jmedchem.6b00460. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299569
78 results